Amsterdam Molecular Therapeutics (Euronext: AMT) a world leader in gene
therapy, today reported positive preclinical data from a study using an AAV
gene therapy product to lower cholesterol. The data show that a single dose
of the gene therapy carrying a short hairpin RNA to silence Apolipoprotein
B100 (ApoB100), resulted in a reduction of serum cholesterol of approximately
80%, without any signs of toxicity. These data validate AMT's technology
platform as a powerful tool for successful gene silencing within target
cells. The data were presented by Annemart Koorneef, scientist at AMT,
yesterday during the 13th Annual American Society of Gene and Cell Therapy
(ASGCT) Meeting in Washington, DC.

"With just a single dose of our cholesterol targeting gene therapy, a
long-lasting, significant reduction of serum cholesterol is achieved. This
preliminary study suggest that AMT's technology may have overcome one of the
major problems of shRNA therapies, namely efficient and non-toxic
intracellular delivery," noted Jorn Aldag, CEO of Amserdam Molecular
Therapeutics.

In the study, AMT used its proprietary AAV-based platform to efficiently
deliver a shRNA that silences both human and mouse Apolipoprotein B100
(ApoB100). A single intravenous administration caused prolonged ApoB100 gene
silencing that was sequence-specific and not associated with liver toxicity,
oversaturation of the cellular miRNA machinery or induction of immune
responses. In addition, it was shown that the shRNA constructs against
ApoB100 specifically and efficiently silence human ApoB100 ex vivo.

ApoB100 is the structural protein of Low Density Lipoprotein (LDL)
particles that carry cholesterol. Silencing of ApoB100 with shRNAs results in
a reduction of LDL-cholesterol and has the potential to be used to treat
hypercholesterolaemia and cardiovascular disease.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in the
development of human gene based therapies. Using adeno-associated viral (AAV)
vectors as the delivery vehicle of choice for therapeutic genes, the company
has been able to design and validate what is probably the first stable and
scalable AAV production platform. This safe and efficacious proprietary
platform offers a unique manufacturing capability which can be applied to a
large number of rare (orphan) diseases that are caused by one faulty gene.
Currently, AMT has a product pipeline with several AAV-based gene therapy
products in LPL Deficiency, Hemophilia B, Duchenne Muscular Dystrophy, Acute
Intermittent Porphyria and Parkinson's Disease at different stages of
research or development.

AMT's lead product, Glybera, for the treatment of lipoprotein lipase
deficiency has been filed for market authorization with the EMA. The current
technology enables AMT to further expand its strategy to develop long-lasting
therapies for severe metabolic diseases.

Certain statements in this press release are "forward-looking statements"
including those that refer to management's plans and expectations for future
operations, prospects and financial condition. Words such as "strategy,"
"expects," "plans," "anticipates," "believes," "will," "continues,"
"estimates," "intends," "projects," "goals," "targets" and other words of
similar meaning are intended to identify such forward-looking statements.
Such statements are based on the current expectations of the management of
Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on
these statements because, by their nature, they are subject to known and
unknown risks and can be affected by factors that are beyond the control of
AMT. Actual results could differ materially from current expectations due to
a number of factors and uncertainties affecting AMT's business, including,
but not limited to, the timely commencement and success of AMT's clinical
trials and research endeavors, delays in receiving U.S. Food and Drug
Administration or other regulatory approvals (i.e. EMEA, Health Canada),
market acceptance of AMT's products, effectiveness of AMT's marketing and
sales efforts, development of competing therapies and/or technologies, the
terms of any future strategic alliances, the need for additional capital, the
inability to obtain, or meet, conditions imposed for required governmental
and regulatory approvals and consents. AMT expressly disclaims any intent or
obligation to update these forward-looking statements except as required by
law. For a more detailed description of the risk factors and uncertainties
affecting AMT, refer to the prospectus of AMT's initial public offering on
June 20, 2007, and AMT's public announcements made from time to time.

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